Skip to main content
Erschienen in:

21.09.2020 | short review

Autologous stem cell transplantation in light-chain amyloidosis

verfasst von: PD Dr. Alexandra Böhm

Erschienen in: memo - Magazine of European Medical Oncology | Ausgabe 1/2021

Einloggen, um Zugang zu erhalten

Summary

Treatment of patients with light-chain amyloidosis (AL amyloidosis) has changed over the last 20 years, and early mortality rates have decreased with prolongation of survival. However, many patients are not cured with conventional therapy. Therefore, all patients should be assessed at diagnosis to determine eligibility for autologous stem cell transplantation (ASCT) since high-dose melphalan and ASCT have been shown to induce long-term hematologic and clinical responses with treatment-related mortality (TRM) <5%. Earlier diagnosis, improvement of supportive care, and deeper remissions with recently available first-line therapy have substantially reduced TRM. However, procedures and experience in complex AL amyloidosis patients still vary between transplant centers because a multidisciplinary team is necessary for an individual risk-adapted approach.
Literatur
1.
Zurück zum Zitat Comenzo RL, Vosburgh E, Simms RW, Bergethon P, Sarnacki D, Finn K, et al. Dose-intensive melphalan with blood stem cell support for the treatment of AL amyloidosis: one-year follow-up in five patients. Blood. 1996;88:2801–6.CrossRef Comenzo RL, Vosburgh E, Simms RW, Bergethon P, Sarnacki D, Finn K, et al. Dose-intensive melphalan with blood stem cell support for the treatment of AL amyloidosis: one-year follow-up in five patients. Blood. 1996;88:2801–6.CrossRef
2.
Zurück zum Zitat Passweg JR, Baldomero H, Bader P, Bonini C, Duarte RF, Dufour C, et al. Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report. Bone Marrow Transplant. 2017;52:811–7.CrossRef Passweg JR, Baldomero H, Bader P, Bonini C, Duarte RF, Dufour C, et al. Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report. Bone Marrow Transplant. 2017;52:811–7.CrossRef
3.
Zurück zum Zitat Dispenzieri A, Kyle RA, Lacy MQ, Therneau TM, Larson DR, Plevak MF, et al. Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study. Blood. 2004;103:3960–3.CrossRef Dispenzieri A, Kyle RA, Lacy MQ, Therneau TM, Larson DR, Plevak MF, et al. Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study. Blood. 2004;103:3960–3.CrossRef
4.
Zurück zum Zitat Gertz MA, Lacy MQ, Dispenzieri A, Hayman SR, Kumar SK, Dingli D, et al. Autologous stem cell transplant for immunoglobulin light chain amyloidosis: a status report. Leuk Lymphoma. 2010;51:2181–7.CrossRef Gertz MA, Lacy MQ, Dispenzieri A, Hayman SR, Kumar SK, Dingli D, et al. Autologous stem cell transplant for immunoglobulin light chain amyloidosis: a status report. Leuk Lymphoma. 2010;51:2181–7.CrossRef
5.
Zurück zum Zitat Rosengren S, Mellqvist UH, Nahi H, Forsberg K, Lenhoff S, Strömberg O, et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994–2009. Bone Marrow Transplant. 2016;51:1569–72.CrossRef Rosengren S, Mellqvist UH, Nahi H, Forsberg K, Lenhoff S, Strömberg O, et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation in Sweden, long-term results from all patients treated in 1994–2009. Bone Marrow Transplant. 2016;51:1569–72.CrossRef
6.
Zurück zum Zitat Cohen AD, Zhou P, Chou J, Teruya-Feldstein J, Reich L, Hassoun H, et al. Risk-adapted autologous stem cell transplantation with adjuvant dexamethasone +/− thalidomide for systemic light-chain amyloidosis: results of a phase II trial. Br J Haematol. 2007;139:224–33.CrossRef Cohen AD, Zhou P, Chou J, Teruya-Feldstein J, Reich L, Hassoun H, et al. Risk-adapted autologous stem cell transplantation with adjuvant dexamethasone +/− thalidomide for systemic light-chain amyloidosis: results of a phase II trial. Br J Haematol. 2007;139:224–33.CrossRef
7.
Zurück zum Zitat Moreau P, Leblond V, Bourquelot P, Facon T, Huynh A, Caillot D, et al. Prognostic factors for survival and response after high-dose therapy and autologous stem cell transplantation in systemic AL amyloidosis: a report on 21 patients. Br J Haematol. 1998;101:766–9.CrossRef Moreau P, Leblond V, Bourquelot P, Facon T, Huynh A, Caillot D, et al. Prognostic factors for survival and response after high-dose therapy and autologous stem cell transplantation in systemic AL amyloidosis: a report on 21 patients. Br J Haematol. 1998;101:766–9.CrossRef
8.
Zurück zum Zitat Sorror ML, Maris MB, Storb R, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood. 2005;106:2912–9.CrossRef Sorror ML, Maris MB, Storb R, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood. 2005;106:2912–9.CrossRef
9.
Zurück zum Zitat Berro M, Arbelbide JA, Rivas MM, Basquiera AL, Ferini G, Vitriu A, et al. Hematopoietic cell transplantation-specific comorbidity index predicts morbidity and mortality in autologous stem cell transplantation. Biol Blood Marrow Transplant. 2017;23:1646–50.CrossRef Berro M, Arbelbide JA, Rivas MM, Basquiera AL, Ferini G, Vitriu A, et al. Hematopoietic cell transplantation-specific comorbidity index predicts morbidity and mortality in autologous stem cell transplantation. Biol Blood Marrow Transplant. 2017;23:1646–50.CrossRef
10.
Zurück zum Zitat Saad A, Mahindra A, Zhang M, Zhong X, Costa LJ, Dispenzieri A, et al. Hematopoietic cell transplant comorbidity index is predictive of survival after autologous hematopoietic cell transplantation in multiple myeloma. Biol Blood Marrow Transplant. 2014;20:402–408.e1.CrossRef Saad A, Mahindra A, Zhang M, Zhong X, Costa LJ, Dispenzieri A, et al. Hematopoietic cell transplant comorbidity index is predictive of survival after autologous hematopoietic cell transplantation in multiple myeloma. Biol Blood Marrow Transplant. 2014;20:402–408.e1.CrossRef
11.
Zurück zum Zitat Gertz MA, Lacy MQ, Dispenzieri A, Kumar SK, Dingli D, Leung N, et al. Refinement in patient selection to reduce treatment-related mortality from autologous stem cell transplantation in amyloidosis. Bone Marrow Transplant. 2013;48:557–61.CrossRef Gertz MA, Lacy MQ, Dispenzieri A, Kumar SK, Dingli D, Leung N, et al. Refinement in patient selection to reduce treatment-related mortality from autologous stem cell transplantation in amyloidosis. Bone Marrow Transplant. 2013;48:557–61.CrossRef
12.
Zurück zum Zitat Skinner M, Sanchorawala V, Seldin DC, Dember LM, Falk RH, Berk JL, et al. High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8‑year study. Ann Intern Med. 2004;140:85–93.CrossRef Skinner M, Sanchorawala V, Seldin DC, Dember LM, Falk RH, Berk JL, et al. High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8‑year study. Ann Intern Med. 2004;140:85–93.CrossRef
13.
Zurück zum Zitat Girnius S, Seldin DC, Meier-Ewert HK, Sloan JM, Quillen K, Ruberg FL, et al. Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement. Bone Marrow Transplant. 2014;49:434–9.CrossRef Girnius S, Seldin DC, Meier-Ewert HK, Sloan JM, Quillen K, Ruberg FL, et al. Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement. Bone Marrow Transplant. 2014;49:434–9.CrossRef
14.
Zurück zum Zitat D’Souza A, Dispenzieri A, Wirk B, Zhang MJ, Huang J, Gertz MA, et al. Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a center for international blood and marrow transplant research study. J Clin Oncol. 2015;33:3741–9.CrossRef D’Souza A, Dispenzieri A, Wirk B, Zhang MJ, Huang J, Gertz MA, et al. Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a center for international blood and marrow transplant research study. J Clin Oncol. 2015;33:3741–9.CrossRef
15.
Zurück zum Zitat Sidana S, Sidiqi MH, Dispenzieri A, Buadi FK, Lacy MQ, Muchtar E, et al. Fifteen year overall survival rates after autologous stem cell transplantation for AL amyloidosis. Am J Hematol. 2019;94:1020–6.CrossRef Sidana S, Sidiqi MH, Dispenzieri A, Buadi FK, Lacy MQ, Muchtar E, et al. Fifteen year overall survival rates after autologous stem cell transplantation for AL amyloidosis. Am J Hematol. 2019;94:1020–6.CrossRef
16.
Zurück zum Zitat Sanchorawala V, Sun F, Quillen K, Sloan JM, Berk JL, Seldin DC. Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation: 20-year experience. Blood. 2015;126:2345–7.CrossRef Sanchorawala V, Sun F, Quillen K, Sloan JM, Berk JL, Seldin DC. Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation: 20-year experience. Blood. 2015;126:2345–7.CrossRef
17.
Zurück zum Zitat Cibeira MT, Sanchorawala V, Seldin DC, Quillen K, Berk JL, Dember LM, et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. Blood. 2011;118:4346–52.CrossRef Cibeira MT, Sanchorawala V, Seldin DC, Quillen K, Berk JL, Dember LM, et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. Blood. 2011;118:4346–52.CrossRef
18.
Zurück zum Zitat Gertz MA, Lacy MQ, Dispenzieri A, Buadi FK, Dingli D, Hayman SR, et al. Stem cell transplantation compared with melphalan plus dexamethasone in the treatment of immunoglobulin light-chain amyloidosis. Cancer. 2016;122:2197–205.CrossRef Gertz MA, Lacy MQ, Dispenzieri A, Buadi FK, Dingli D, Hayman SR, et al. Stem cell transplantation compared with melphalan plus dexamethasone in the treatment of immunoglobulin light-chain amyloidosis. Cancer. 2016;122:2197–205.CrossRef
19.
Zurück zum Zitat Abdallah N, Sidana S, Dispenzieri A, Lacy M, Buadi F, Hayman S, et al. Outcomes with early vs. deferred stem cell transplantation in light chain amyloidosis. Bone Marrow Transplant. 2020;55:1297–1304.CrossRef Abdallah N, Sidana S, Dispenzieri A, Lacy M, Buadi F, Hayman S, et al. Outcomes with early vs. deferred stem cell transplantation in light chain amyloidosis. Bone Marrow Transplant. 2020;55:1297–1304.CrossRef
20.
Zurück zum Zitat Kourelis TV, Kumar SK, Gertz MA, Lacy MQ, Buadi FK, Hayman SR, et al. Coexistent multiple myeloma or increased bone marrow plasma cells define equally high-risk populations in patients with immunoglobulin light chain amyloidosis. J Clin Oncol. 2013;31:4319–24.CrossRef Kourelis TV, Kumar SK, Gertz MA, Lacy MQ, Buadi FK, Hayman SR, et al. Coexistent multiple myeloma or increased bone marrow plasma cells define equally high-risk populations in patients with immunoglobulin light chain amyloidosis. J Clin Oncol. 2013;31:4319–24.CrossRef
21.
Zurück zum Zitat Al Saleh AS, Sidiqi MH, Sidana S, Muchtar E, Dispenzieri A, Dingli D, et al. Impact of consolidation therapy post autologous stem cell transplant in patients with light chain amyloidosis. Am J Hematol. 2019;94:1066–71.CrossRef Al Saleh AS, Sidiqi MH, Sidana S, Muchtar E, Dispenzieri A, Dingli D, et al. Impact of consolidation therapy post autologous stem cell transplant in patients with light chain amyloidosis. Am J Hematol. 2019;94:1066–71.CrossRef
22.
Zurück zum Zitat Minnema MC, Nasserinejad K, Hazenberg B, Hegenbart U, Vlummens P, Ypma PF, et al. Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial. Haematologica. 2019;104:2274–82.CrossRef Minnema MC, Nasserinejad K, Hazenberg B, Hegenbart U, Vlummens P, Ypma PF, et al. Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial. Haematologica. 2019;104:2274–82.CrossRef
23.
Zurück zum Zitat Wechalekar AD, Hawkins PN, Gillmore JD. Perspectives in treatment of AL amyloidosis. Br J Haematol. 2008;140:365–77.CrossRef Wechalekar AD, Hawkins PN, Gillmore JD. Perspectives in treatment of AL amyloidosis. Br J Haematol. 2008;140:365–77.CrossRef
24.
Zurück zum Zitat Gertz MA, Lacy MQ, Dispenzieri A, Ansell SM, Elliott MA, Gastineau DA, et al. Risk-adjusted manipulation of melphalan dose before stem cell transplantation in patients with amyloidosis is associated with a lower response rate. Bone Marrow Transplant. 2004;34:1025–31.CrossRef Gertz MA, Lacy MQ, Dispenzieri A, Ansell SM, Elliott MA, Gastineau DA, et al. Risk-adjusted manipulation of melphalan dose before stem cell transplantation in patients with amyloidosis is associated with a lower response rate. Bone Marrow Transplant. 2004;34:1025–31.CrossRef
25.
Zurück zum Zitat Tandon N, Muchtar E, Sidana S, Dispenzieri A, Lacy MQ, Dingli D, et al. Revisiting conditioning dose in newly diagnosed light chain amyloidosis undergoing frontline autologous stem cell transplant: impact on response and survival. Bone Marrow Transplant. 2017;52:1126–32.CrossRef Tandon N, Muchtar E, Sidana S, Dispenzieri A, Lacy MQ, Dingli D, et al. Revisiting conditioning dose in newly diagnosed light chain amyloidosis undergoing frontline autologous stem cell transplant: impact on response and survival. Bone Marrow Transplant. 2017;52:1126–32.CrossRef
26.
Zurück zum Zitat Tandon N, Muchtar E, Sidana S, Dispenzieri A, Lacy MQ, Dingli D, Buadi FK, Hayman SR, Chakraborty R, Hogan WJ, Gonsalves W, Warsame R, Kourelis TV, Leung N, Kapoor P, Kumar SK, Gertz MA. Revisiting conditioning dose in newly diagnosed light chain amyloidosis undergoing frontline autologous stem cell transplant: impact on response and survival. Bone Marrow Transplant. 2017; 52(8):1126–32.CrossRef Tandon N, Muchtar E, Sidana S, Dispenzieri A, Lacy MQ, Dingli D, Buadi FK, Hayman SR, Chakraborty R, Hogan WJ, Gonsalves W, Warsame R, Kourelis TV, Leung N, Kapoor P, Kumar SK, Gertz MA. Revisiting conditioning dose in newly diagnosed light chain amyloidosis undergoing frontline autologous stem cell transplant: impact on response and survival. Bone Marrow Transplant. 2017; 52(8):1126–32.CrossRef
Metadaten
Titel
Autologous stem cell transplantation in light-chain amyloidosis
verfasst von
PD Dr. Alexandra Böhm
Publikationsdatum
21.09.2020
Verlag
Springer Vienna
Erschienen in
memo - Magazine of European Medical Oncology / Ausgabe 1/2021
Print ISSN: 1865-5041
Elektronische ISSN: 1865-5076
DOI
https://doi.org/10.1007/s12254-020-00644-7

Weitere Artikel der Ausgabe 1/2021

memo - Magazine of European Medical Oncology 1/2021 Zur Ausgabe